CreatorsPublishersAdvertisers
View more in
Diseases & Treatments

Gene therapy restores missing enzyme in rare childhood disease

NIH Director's Blog
 2021-07-26

Cover picture for the articleA small study found that gene therapy delivered into the brain was safe and beneficial in treating a debilitating neurological disease. The results show promise for treating AADC deficiency and other genetic diseases with gene therapies. Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that leads to...

www.nih.gov

Comments / 0

Comments / 0